Therapeutic base editing alleviates restrictive cardiomyopathy - PubMed
3 hours ago
- #restrictive cardiomyopathy
- #base editing
- #AAV
- Restrictive cardiomyopathy (RCM) is a severe cardiac disorder caused by mutations in sarcomeric proteins, particularly the TNNI3 gene (e.g., p.R192H).
- A murine model with the Tnni3R193H mutation was developed to mimic human RCM.
- Adenine base editor (ABE) delivered via adeno-associated virus (AAV) effectively corrected the Tnni3R193H mutation in adult RCM mice.
- The correction led to significant improvement in cardiac functions.
- Base editing is established as a therapeutic strategy for RCM and other genetic cardiomyopathies.